Module232025

12/05/2025

Paediatric Challenges

Lack of research

Lack clinical drug development expertise

Lack of age appropriate formulations

Children are not small adults

Limited marketing potential

Ethical concerns

Adverse events- can occur late. Long term Follow up needed

Difficult to organize trials,

Higher cost programs

The Organisation for Professionals in Regulatory Affairs

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EU vs US

Key Difference

EU

USA

PIP submission

Before end of human PK studies. Not after initiation of Phase 3 studies All conditions within the MedDRA Higher Level Term (HLT) of the proposed adult indication. Neonates: - pre-term newborns - term newborns Adolescents:12 - 18 years

Before initiation of paediatric investigations or <60 days from End of Phase 2 Only indication proposed ODD/ adult-only conditions are exempt Neonate = 1 category 12 to <17 years in the US

EU

Indication Scope

Global Development

US

Age Subsets

https://boydconsultants.com/paediatric-development harmonisation-in-europe-and-the-us-myth-or-reality/

The Organisation for Professionals in Regulatory Affairs

4